Chief Complaint/History of Present Illness:
Phenylketonuria (PKU) is a liver enzyme deficiency or inability to metabolize the essential amino acid, phenylalanine (phe). The accumulation of phe results in severe, neurological toxicities and mental retardation. Diagnosis through newborn screening necessitates early and continuous treatment with a phe-restricted diet and provision of medical food. There is no cure.
What are Medical Foods?
According to 21 USC 360ee(b)(3)), medical food is defined as “a food which is formulated to be consumed or administered enterally under the supervision of a physician and which is intended for the specific dietary management of a disease or condition for which distinctive nutritional requirements, based on recognized scientific principles, are established by medical evaluation." And, as per the FDA on Regulation of Medical Foods (1996 ANPR), it has been recognized “although Congress provided a statutory definition for medical foods, the legislative history of the Orphan Drug Amendments does not discuss the definition and, therefore, does not provide any further information regarding the types of products that the definition was intended to cover.1
Despite the provision of clarifying criteria that was later to follow under the 1990 Amendments, history has shown that good intentions have not made for good policy. The impact for lack of a strict standard as intended is still felt today and much reflected by broad, loose interpretation and application of the rules; thereby rendering the federal definition of medical foods a nonworking definition. Given the recent trends in patient focused drug development and patient-centered medicine, I would like to draw attention to the sociological implications from the patient’s perspective on FDA framework, which can be characteristically defined as satisficing, or “making the best decision under constraints related to time, information, and other resources.2
New Strategy Needed
I have modeled my policy analysis after a pharmaceutical care short-plan to spark a paradigm shift in thinking from traditional approach and in mapping out selection of a new proposed approach that gives emphasis to the drug properties of medical foods. Ethical leadership should engage and empower the patient in the development of their own pharmacotherapeutic care, - tailored to needs, especially when policies have a direct impact on their own welfare and safety.
In contrast, it has been my experience, as in the case for others, that the government has found a favored policy option in affording incentive to those who engage in a game of regulatory risk through restricted competition. The lucrative gains from malfeasance for a medical foods market dominated by chronic, prevalent diseases comes at the expense of a very vulnerable subpopulation of individuals living with (PKU) and similar inborn errors, for which the category of medicals foods was originally engineered. A summary on transformative legislation is incorporated in the detailed account, Solving the Satisficing Cost of Medical Food Misbranded
in order to set the stage for understanding governmental behaviors and the resulting series of damaging, repeated patterns of events that are of historical significance and much compounded by the inefficiency of traditional drug development paradigms on orphan therapies today.
In contrast to lengthy, legislative processes by politicians who “use science the ways drunks use lampposts – for support, not for illumination,3
” my efforts target regulatory framework and seek to recalibrate the system’s seemingly long standing “punctuated equilibrium4
” with proposed responsive regulation, not tighter regulation. In defense of a new paradigm for medical foods, it is argued the FDA has surrendered to a fallacy of belief in continually denying the proven, legitimate, safe and beneficial uses of medical foods under such narrow constraint of federal statute because of the negative outcomes posed by a few. Although the problems surrounding the category of medical foods and one-size-fits-all approach have reached the identification stage in the policy process, new risk has materialized since 2003 withdrawal of the ANPR, thereby throwing a wrench into regulatory gears and signaling an reaffirmation of truth that abandonment for an age-old traditional approach is justified.
Solving the Satisficing Cost of Medical Food Misbranded
offers a reality check in the legendary saga of medical foods regime that can best be delivered from real-life experiences as told from the front line experts. The patient experience spans noble, pan-disciplinary efforts, which have been rendered essentially futile due to systematic failures and a government that works against the people. Access challenges include prescribers who have lost their prescribing power because options are dictated by third parties. Patients have no equitable, affordable means to treatment because the current system (HCPCS) does not even recognize or reflect the safe and appropriate use of medical foods as distinct from weight reduction products, dietary supplements, and other nondrug products. And, legitimate, small orphan innovators are constrained in the shadows of big market players that cater to a larger population with more prevalent diseases that unlike PKU, have many FDA approved drugs as treatment options. Furthermore, the policy shortcomings are much evidenced by variations in state policy and a Congressional report card punctuated with transformative legislation and recent initiatives like 21st
Century Cures that afford no critical consumer protections to the PKU community to assure access or mitigate the accumulating risk and chronology of damage documented by medical establishment.
It is my position that the promulgation of safety is a shared responsibility and the ideas introduced in the full, detailed report are intended to return the system back to synchronicity with aligning patient-specific needs and the leadership tasked to safeguard and shepherd the health of all Americans. The innovative thinking behind the proposed methodology is a simple, practical, and feasible alternative approach. Risk is simply eliminated by removing the misbranding with an exceptional labeling standard predominantly used in the most widely accepted policy language known to the pharmaceutical world today, the National Drug Code nomenclature system.
To access full text of the article, author can be reached via the contact page at www.pkupioneer.com or emailed at firstname.lastname@example.org
Note: Solving the Satisficing Cost of Medical Food Misbranded (2014)
serves as an introductory paper to subsequent, in-depth analysis and completion of strategic capstone project report, FDA Regime on Medical Foods for Phenylketonuria: Promoting Market Viability through Retroadaptive Licensing of the National Drug Code (2015)
. Policy analysis and final project submissions were made to New England College in partial fulfillment of the requirements for degree of Master of Arts in Public Policy.
FDA, US Department of Health and Human Services, 21 CFR Ch 1 [Docket No. 96-N-0364], Federal Register, vol. 61 No. 231, November 29, 1996, p60662
Birkland, Thomas A. An Introduction to the Policy Process, 3rd Edition, 2011 ME Sharpe, Inc. p302
Ibid at 2, p295
Ibid at 2, p300 quoted in Frank R. Baumgartner and Bryan D. Jones, Agendas and Instability in American Politics.